TriangleNCT00465985 · CITED IN SOURCE DOCUMENTS
Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE3
- Status at capture
- COMPLETED
- Registry last update
- 2017-08-28
This study is designed to provide efficacy and safety data for ACZ885 (a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody) administered as an injection subcutaneously (s.c.) in patients with Muckle-Wells Syndrome. Part I is an 8-week open-label, active treatment period to identify ACZ885 responders. Part II is a double-blind, placebo-controlled period to assess primarily the efficacy of ACZ885 compared to placebo. Part III is an open-label, active treatment period where patients will receive ACZ885 every 8 weeks after withdrawal or completion of Part II.
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What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part)
Number of Participants With Treatment Response in Part I (After 8 Weeks)
Investigator's Clinical Assessment of Autoinflammatory Disease Activity & Participant's Assessment of Symptoms at End of Part II (After 24 Weeks of the Double-blind Part)
Change in Inflammation Markers at the End of Part II (C-reactive Protein and/or Serum Amyloid A) (After 24 Weeks of the Double-blind Part) From Week 8.
Pharmacokinetics (CLD (L/d))
Number of Participants Who Experienced a Disease Flare in Part II
Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part I.
Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part II.
Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part III.
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- Inclusion Criteria: * Molecular diagnosis of NALP3 mutations and clinical picture resembling Muckle-Wells Syndrome. * Muckle-Wells Syndrome patients who participated in the CACZ885A2102 study, will have the option to participate in this study upon disease flare * Muckle-Wells Syndrome patients requiring medical intervention either untreated or treated (i.e. under ACZ885, anakinra, or any other investigational IL-1 blocking therapy). Exclusion Criteria: * History of being immunocompromised, including a positive HIV at screening test result. * No live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose. * History of significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial. * History of recurrent and/or evidence of active bacterial, fungal, or viral infections. * Positive tuberculin skin test at 48 to 72 hours after administration at the screening visit or within 2 months prior to the screening visit, according to national guidelines. Other protocol-defined inclusion/exclusion criteria may apply
- healthy Volunteers
- false
- maximum Age
- 75 Years
- minimum Age
- 4 Years
- sex
- ALL
- std Ages
- CHILD
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- intervention Names
- Drug: ACZ885
- label
- Part I, Part II-arm1, & Part III
- type
- EXPERIMENTAL
- intervention Names
- Drug: Placebo
- label
- Part II - arm 2
- type
- PLACEBO_COMPARATOR
- interventions
- arm Group Labels
- Part I, Part II-arm1, & Part III
- name
- ACZ885
- type
- DRUG
- arm Group Labels
- Part II - arm 2
- name
- Placebo
- type
- DRUG
Study design
- allocation
- RANDOMIZED
- intervention Model
- PARALLEL
- masking Info
- masking
- QUADRUPLE
- who Masked
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
- primary Purpose
- TREATMENT
Enrollment
- count
- 35
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- Determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. Data expressed as a percent of participants who had experienced a flare by the end of Part II.
- measure
- Percent of Participants With Disease Flare in Part II (After 24 Weeks of the Double-blind Part)
- time Frame
- 32 weeks after study start
- description
- Disease flare is determined by the Physician's global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. Disease Flare = the C-reactive protein and/or serum amyloid A (SAA) \> 30 mg/L and either a PGA \> minimal, or PGA equal to minimal and \> minimal SD.
- measure
- Number of Participants Who Experienced a Disease Flare in Part II
- time Frame
- 32 weeks after study start
- secondary Outcomes
- description
- Treatment response was based on Physician's global assessment(PGA) of autoinflammatory disease activity, assessment of skin disease(SD) and serum values of C-reactive protein(CRP) and/or serum amyloid A(SAA). Complete Response (CR):PGA and SD ≤ minimal and normal CRP and/or SAA. Partial Response (PR): a reduction of CRP and/or SAA from baseline (BL) by \>30% but not reaching normal values and PGA improvement from BL by at least one category. Disease flare: a CRP and/or SAA \> 30 mg/L and either PGA \> minimal or PGA = minimal and SD \> minimal. Non-responders = no PR by Day 8 or no CR by Day 15.
- measure
- Number of Participants With Treatment Response in Part I (After 8 Weeks)
- time Frame
- 8 weeks after study start
- description
- A 5-point scale was used for the Physician's global assessment on autoinflammatory disease activity (absent, minimal, mild, moderate and severe) and for the assessment of the following items: * skin disease (urticarial skin rash) * arthralgia * myalgia * headache/migraine * conjunctivitis * fatigue/malaise * other symptoms related to autoinflammatory syndrome * other symptoms not related to autoinflammatory syndrome
- measure
- Investigator's Clinical Assessment of Autoinflammatory Disease Activity & Participant's Assessment of Symptoms at End of Part II (After 24 Weeks of the Double-blind Part)
- time Frame
- 32 weeks after study start
- measure
- Change in Inflammation Markers at the End of Part II (C-reactive Protein and/or Serum Amyloid A) (After 24 Weeks of the Double-blind Part) From Week 8.
- time Frame
- Week 8 and Week 32
- description
- Assessed serum clearance of ACZ885.
- measure
- Pharmacokinetics (CLD (L/d))
- time Frame
- 48 weeks after study start
- measure
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part I.
- time Frame
- until Week 8
- measure
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part II.
- time Frame
- 32 weeks after study start
- measure
- Pharmacodynamics Measured by Interleukin-1β (IL-1β) Concentrations at End of Part III.
- time Frame
- 48 weeks after study start
Full study description
- brief Summary
- This study is designed to provide efficacy and safety data for ACZ885 (a fully human anti-interleukin-1beta (anti-IL-1beta) monoclonal antibody) administered as an injection subcutaneously (s.c.) in patients with Muckle-Wells Syndrome. Part I is an 8-week open-label, active treatment period to identify ACZ885 responders. Part II is a double-blind, placebo-controlled period to assess primarily the efficacy of ACZ885 compared to placebo. Part III is an open-label, active treatment period where patients will receive ACZ885 every 8 weeks after withdrawal or completion of Part II.
Source references
- references
- citation
- Kone-Paut I, Lachmann HJ, Kuemmerle-Deschner JB, Hachulla E, Leslie KS, Mouy R, Ferreira A, Lheritier K, Patel N, Preiss R, Hawkins PN; Canakinumab in CAPS Study Group. Sustained remission of symptoms and improved health-related quality of life in patients with cryopyrin-associated periodic syndrome treated with canakinumab: results of a double-blind placebo-controlled randomized withdrawal study. Arthritis Res Ther. 2011;13(6):R202. doi: 10.1186/ar3535. Epub 2011 Dec 9.
- pmid
- 22152723
- type
- DERIVED
- citation
- Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, Gitton X, Widmer A, Patel N, Hawkins PN; Canakinumab in CAPS Study Group. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med. 2009 Jun 4;360(23):2416-25. doi: 10.1056/NEJMoa0810787.
- pmid
- 19494217
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- Colchicine (Study 3, ILARIS (ACZ885) 150mg) 14.1 Treatment of CAPS The efficacy and safety of ILARIS for the treatment of CAPS was demonstrated in CAPS Study (NCT00465985), a 3-part trial in patients 9 to 74 years of age with the MWS phenotype of CAPS. Throughout the trial, patients weighing more than 40 kg received ILARIS 150 m
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- 53% to 96%. At the end of Part 2, all 15 patients treated with ILARIS had absent or minimal disease activity and skin disease (see Table 4). In a second trial (NCT00465985), patients 4 to 74 years of age with both MWS and FCAS phenotypes of CAPS were treated in an open-label manner. Treatment with ILARIS resulted in clinically si
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Preserved source evidence · Not medical advice